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Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

In an early-phase lentiviral gene therapy trial, gene-corrected autologous hematopoietic stem cells show sustained engraftment and phenotypic correction in non-conditioned patients with Fanconi anemia.

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Main Authors: Rio, Paula (Author) , Navarro, Susana (Author) , Wei, Wang (Author) , Sánchez-Domínguez, Rebeca (Author) , Pujol, Roser M. (Author) , Segovia, José C. (Author) , Bogliolo, Massimo (Author) , Merino, Eva (Author) , Wu, Ning (Author) , Salgado, Rocío (Author) , Lamana, María L. (Author) , Yañez, Rosa M. (Author) , Casado, José A. (Author) , Giménez, Yari (Author) , Román-Rodríguez, Francisco J. (Author) , Álvarez, Lara (Author) , Alberquilla, Omaira (Author) , Raimbault, Anna (Author) , Guenechea, Guillermo (Author) , Lozano, M. Luz (Author) , Cerrato, Laura (Author) , Hernando, Miriam (Author) , Gálvez, Eva (Author) , Hladun, Raquel (Author) , Giralt, Irina (Author) , Barquinero, Jordi (Author) , Galy, Anne (Author) , Andoín, Nagore García de (Author) , López, Ricardo (Author) , Catalá, Albert (Author) , Schwartz, Jonathan D. (Author) , Surrallés, Jordi (Author) , Soulier, Jean (Author) , Schmidt, Manfred (Author) , Heredia, Cristina Díaz de (Author) , Sevilla, Julián (Author) , Bueren, Juan A. (Author)
Format: Article (Journal)
Language:English
Published: 09 September 2019
In: Nature medicine
Year: 2019, Volume: 25, Issue: 9, Pages: 1396-1401
ISSN:1546-170X
DOI:10.1038/s41591-019-0550-z
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/s41591-019-0550-z
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/s41591-019-0550-z
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Author Notes:Paula Río, Susana Navarro, Wei Wang, Rebeca Sánchez-Domínguez, Roser M. Pujol, José C. Segovia, Massimo Bogliolo, Eva Merino, Ning Wu, Rocío Salgado, María L. Lamana, Rosa M. Yañez, José A. Casado, Yari Giménez, Francisco J. Román-Rodríguez, Lara Álvarez, Omaira Alberquilla, Anna Raimbault, Guillermo Guenechea, M. Luz Lozano, Laura Cerrato, Miriam Hernando, Eva Gálvez, Raquel Hladun, Irina Giralt, Jordi Barquinero, Anne Galy, Nagore García de Andoín, Ricardo López, Albert Catalá, Jonathan D. Schwartz, Jordi Surrallés, Jean Soulier, Manfred Schmidt, Cristina Díaz de Heredia, Julián Sevilla, and Juan A. Bueren

MARC

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